A multi-center, open-label, uncontrolled, single-arm, extension study to determine the long-term safety and tolerability of oral lucerastat in adult subjects with Fabry disease.
Fabry disease is a rare inherited condition that causes fatty substances to build up inside the body's cells. Over time, this build-up can affect vital organs such as the kidneys and heart. Lucerastat is an experimental medicine taken by mouth that aims to slow down the production of these fatty substances. This clinical trial is an 'extension study'. This means it follows on from earlier trials to see how safe lucerastat is when taken over months or years. Because the trial is 'open-label', every person who takes part will receive the active medicine; there is no dummy pill (placebo). The main aim is to watch closely for any side effects and learn how comfortably people can take lucerastat in their daily lives. The results will help doctors understand whether this tablet could become a reliable long-term treatment option.
At a glance
What is this study about?
Fabry disease is a rare genetic condition where the body lacks a specific enzyme needed to break down a type of fat. As a result, this fatty material gradually builds up in cells throughout the body, particularly affecting the kidneys, heart, nervous system, and skin. Standard treatments often require regular, lifelong infusions into a vein at a clinic or hospital.
Lucerastat is being developed as an alternative treatment that can be swallowed as a capsule at home. Instead of replacing the missing enzyme, it works by reducing the amount of fat the body produces in the first place. If successful, having an oral treatment could make managing Fabry disease simpler and more flexible for patients.
This study is an extension trial designed to check the long-term safety and tolerability of lucerastat. While earlier trials checked how the drug works over shorter periods, this study monitors participants over an extended period. Researchers want to ensure that taking the medicine daily does not cause harmful or uncomfortable side effects over time.
Because this is a single-arm study, there is no comparison group and no placebo. Everyone who joins will be given lucerastat. By tracking each participant's health closely, doctors hope to gather the clear evidence needed to decide if the medicine is safe for widespread, long-term use.
Key takeaways
- Tests the long-term safety and tolerability of lucerastat, a daily capsule for Fabry disease.
- Open to adults aged 18 and over who have Fabry disease.
- Every participant receives the active study medicine with no dummy pills.
- Involves regular clinic visits for routine health checks, blood tests, and heart monitoring.
- Participation is entirely voluntary and will not impact your normal NHS care.
Who may be eligible?
This trial is designed for adults aged 18 and older who have a confirmed diagnosis of Fabry disease. Both men and women are eligible to take part.
Because this is an extension study, participants are usually people who have already taken part in a previous clinical trial testing lucerastat. The study doctor will check your medical records to confirm whether you meet the criteria to roll over into this long-term phase.
You may not be able to take part if you have certain other severe medical conditions, if you are unable to swallow capsules, or if you are pregnant or breastfeeding. Your study doctor will review all your current treatments to make sure lucerastat is safe for you.
Could this study suit you?
Answer these quick questions to see if you may be eligible. This is a guide only — the research team makes the final call.
- Are you aged 18 or older?
- Do you have a confirmed medical diagnosis of Fabry disease?
- Have you previously completed an earlier clinical trial for lucerastat?
- Are you able to swallow oral capsules daily?
- Are you willing and able to attend scheduled hospital check-ups?
What does participation involve?
If you join this trial, you will be asked to take lucerastat capsules by mouth each day at home. You will need to visit the hospital or study clinic regularly so the medical team can monitor your health. During these visits, doctors and nurses will carry out routine health checks, such as measuring your blood pressure, taking blood and urine samples, and performing heart traces (ECGs). You will also be asked questions about how you are feeling and whether you have noticed any side effects. The study runs over an extended period, often lasting several years, to ensure the medicine remains safe throughout long-term use.
Potential risks and benefits
Locations (7)
- —UnverifiedAustria
- —UnverifiedFrance
- —UnverifiedGermany
- —UnverifiedNorway
- —UnverifiedPoland
- —UnverifiedBelgium
- —UnverifiedSpain
Common questions
What is an extension study?
An extension study allows patients who completed an earlier trial to carry on taking the medicine so doctors can study its long-term safety.
Will I be given a dummy pill (placebo)?
No. This is an open-label study, which means every participant receives the real medicine, lucerastat.
How is lucerastat taken?
Lucerastat is taken orally as capsules swallowed with water, rather than given through a drip or injection.
Does taking part cost anything?
No. The study medicine and all trial-related tests are provided free of charge, and travel expenses to clinic visits are typically reimbursed.
Can I leave the study if I change my mind?
Yes. You have the right to leave the trial at any point, and your usual healthcare team will support your ongoing care.
How to find out more
Always speak to your GP or specialist before deciding to take part in a study.
Interested in taking part?
Discussion
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